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A phase Ib/IIa clinical trial of dantrolene sodium in patients with Wolfram syndrome

BACKGROUND: Wolfram syndrome is a rare ER disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration. Although there is no treatment for Wolfram syndrome, preclinical studies in cell and rodent models suggest that therapeutic strategies targ...

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Detalles Bibliográficos
Autores principales:	Abreu, Damien, Stone, Stephen I., Pearson, Toni S., Bucelli, Robert C., Simpson, Ashley N., Hurst, Stacy, Brown, Cris M., Kries, Kelly, Onwumere, Chinyere, Gu, Hongjie, Hoekel, James, Tychsen, Lawrence, Van Stavern, Gregory P., White, Neil H., Marshall, Bess A., Hershey, Tamara, Urano, Fumihiko
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society for Clinical Investigation 2021
Materias:	Clinical Medicine
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8410026/ https://www.ncbi.nlm.nih.gov/pubmed/34185708 http://dx.doi.org/10.1172/jci.insight.145188

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